Retrophin drug to treat nervous system disorder fails late-stage study

[August 22, 2019]  (Reuters) - Retrophin Inc said on Thursday its treatment for a rare neurological disorder failed to meet the main goal of improving patient's ability to conduct daily activities in a late-stage study.

The treatment, fosmetpantotenate, was being tested in 84 patients with pantothenate kinase-associated neurodegeneration, a rare, genetic and life-threatening disorder.

The condition, which can lead to movement disorders and visual impairment, is estimated to affect up to 5,000 people worldwide, Retrophin said.

The study analyzed fosmetpantotenate using a scale to measure improvement in daily activity in patients called the PKAN-ADL scale through a period of 24 weeks.

The company said fosmetpantotenate was found to be safe and well-tolerated and that it would work closely with the study investigators to further analyze the results.

The treatment, tested against placebo, also failed to meet its secondary goal, the company said.

(Reporting by Aakash Jagadeesh Babu in Bengaluru; Editing by Shinjini Ganguli)

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